Limited coverage drugs – sapropterin dihydrochloride

sapropterin dihydrochloride

100 mg tablet

100 mg and 500 mg sachet with powder for oral solution

Initial coverage

For the treatment of hyperphenylalaninemia (HPA) due to tetrahydrobiopterin-responsive (BH4-responsive) phenylketonuria (PKU) in patients who meet ALL of the following initiation criteria:

Confirmed diagnosis of phenylketonuria (PKU) AND managed by a physician specialized in metabolic/biochemical diseases

AND

Adherence to low-protein diet and formulas AND baseline protein intake assessment by a dietitian

AND

Baseline blood phenylalanine (Phe) levels > 360 µmol/L despite adherence to a low-protein diet (requires at least 2 baseline levels during the preceding  3- to 6-month time frame)

AND

Ability to adhere to medication regimen

AND

Special Authority request is submitted by a physician specialized in metabolic/biochemical diseases

6 months

Renewal coverage

For the treatment of HPA due to BH4-responsive PKU in patients who meet ALL of the following renewal criteria:

Adherence to low-protein diet and formulas and sapropterin, AND followed by a dietitian

AND

Phe levels meet treatment targets (at least 2 levels measured at least 1 month apart):

• Normal sustained blood Phe levels < 360 μmol/L OR
• Sustained blood Phe reduction of at least 30% compared to baseline if the Phe baseline level was < 1200 μmol/L OR
• Sustained blood Phe reduction of at least 50% compared to baseline if the Phe baseline level was > 1200 μmol/L

AND

Demonstrated increase of dietary protein tolerance based on targets set between the clinician and patient AND managed by a physician specialized in metabolic/biochemical diseases

AND

Special Authority request is submitted by a physician specialized in metabolic/biochemical diseases

1 year

Pregnant patients may qualify for coverage upon meeting the following modified criteria:

Diagnosis of phenylketonuria (PKU) confirmed through an approved test and managed by a physician specialized in metabolic/biochemical diseases

AND

Baseline blood phenylalanine (Phe) levels > 360 µmol/L despite adherence with all recommendations for dietary intervention and monitoring. Pregnant patients only require demonstration of one baseline Phe level > 360 µmol/L despite compliance with low-protein diet

AND

Special Authority request is submitted by a physician specialized in metabolic/biochemical diseases

1 year